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Biliary Tract CancerSeptember 2020

What the ROAR Basket Trial Found — Dabrafenib + Trametinib for BRAF-Mutant Bile Duct Cancer

ROAR tested dabrafenib plus trametinib — two daily targeted pills — in 43 people with advanced BRAF-mutant biliary tract cancer who had failed prior chemotherapy. Tumors shrank in about half.

What the trial was testing

The ROAR enrolled 206 patients with biliary tract cancer. The study was sponsored by Novartis Pharmaceuticals and tracked outcomes across the full group of patients who matched the trial's eligibility profile.

It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.

What the results showed

About 50% tumor response rate in BRAF-mutant biliary tract cancer.

The Lancet Oncology · 2020 · NCT02034110

These findings — that in advanced BRAF-mutant biliary tract cancer on dabrafenib plus trametinib — were published in the The Lancet Oncology and represent the headline result of the study.

Researchers tracked outcomes across 206 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.

What this means for patients

For patients with biliary tract cancer, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.

What you can do now

Dabrafenib (Tafinlar) plus trametinib (Mekinist) is FDA-approved for any solid tumor with a BRAF V600E mutation, including biliary tract cancer, and available now. Ask an oncologist about BRAF testing on your tumor — most centers test routinely for advanced bile duct cancer.

Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.